Human Stem Cell Use: Adult or Tissue Stem Cell

Curing Sickle cell Disease with CRISPR-Cas9 genome editing

Translational Candidate The principal objective of this program is to bring a Cas9-based gene editing cure for sickle cell disease to the pre-IND stage of development. Area of Impact The principal barriers to transplant for SCD are lack of a donor and the toxicity of transplant, which can be overcome by the Cas9-based approach Mechanism […]

An autologous somatic stem cell therapy for the treatment of osteonecrosis

Translational Candidate An autologous somatic stem cell therapy for the treatment of osteonecrosis. Area of Impact Osteonecrosis is a painful, progressive disease for which there is no treatment, save replacing the dead bone with a metal implant. Mechanism of Action Autografts contain skeletal stem cells. In young patients, these stem cells differentiate and give rise […]

Placental Derived Natural Killer Cells to Target Solid Tumor Cancer Stem Cells (CSC)

Translational Candidate Placental-derived stem cells becoming a unique NK-like cell Area of Impact solid tissue cancers Mechanism of Action These expanded cells share many characteristics as NK cells but appear to represent a unique stage in differentiation and a cell-type with advantageous properties. These cells have been demonstrated to persist for much longer periods of […]

Stem Cell-Based iNKT Cell Therapy for Cancer

Translational Candidate Lenti/iNKT-sr39TK Modified Autologous Human CD34+ Hematopoietic Stem Cells (HSCs) Area of Impact The targeted area of impact for the candidate is cancer therapy, in particular cancers that are lacking existing effective treatments. Mechanism of Action The proposed candidate will generate therapeutic levels of invariant natural killer T (iNKT) cells in cancer patients, helping […]

Overexpression of HexA/HexB by lentivector expression in blood cells to treat Tay-Sachs and Sandhoff disease

Translational Candidate Autologous hematopoietic stem cells transduced with a lentiviral vector expressing wild type human HexA and HexB. Area of Impact The therapeutic candidate would halt disease progression in Tay-Sachs and Sandhoff disease patients who have no curative or ameliorating treatment. Mechanism of Action Wild type HexA and HexB will be delivered to affected neurons […]

RPESC-RPE-4W Therapy for dry Age-related Macular Degeneration

Therapeutic Candidate or Device Retina pigment epithelial stem cell (RPESC)-derived RPE progeny at 4 weeks of differentiation (RPESC-RPE-4W) Indication Dry age-related macular degeneration (dry AMD). Therapeutic Mechanism Dry AMD involves loss of the RPE cells that support the overlying retina and vision. We transplant laboratory grown RPESC-RPE-4W cell to replace the RPE cells lost in […]

The CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele

Therapeutic Candidate or Device Allogeneic Placenta-derived Mesenchymal Stem Cells Seeded on Cook Biodesign® Dural Graft Extracellular Matrix (PMSC-ECM) Indication Myelomeningocele (MMC) -or Spina Bifida -diagnosed prenatally Therapeutic Mechanism Placenta-derived mesenchymal stem cells (PMSCs) act by a paracrine mechanism, secreting a variety of growth factors, cytokines, and extracellular vesicles. This secretory profile is unique to PMSCs […]

Phase I Trial of Locoregionally Delivered Autologous B7-H3 CAR T Cells (B7-H3CART) in Adults with Recurrent Glioblastoma Multiforme

Therapeutic Candidate or Device Autologous T cells genetically engineered to express a Chimeric Antigen Receptor targeting B7-H3 (B7-H3CART) Indication Brain tumors in adults: Glioblastoma Multiforme (GBM) Therapeutic Mechanism Progenitor B7-H3CART cells will recognize GBM cancer cells expressing B7-H3, become activated, divide, and kill the cancer cells Unmet Medical Need Glioblastoma Multiforme (GBM) is the most […]

Phase 2b Clinical Study of KPI-012 Topical Ophthalmic Human Mesenchymal Stem Cell Secretome for the Treatment of Persistent Corneal Epithelial Defect

Therapeutic Candidate or Device KPI-012 is a human bone marrow mesenchymal stem cell secretome formulated as a topical ophthalmic therapeutic Indication Treatment of persistent corneal epithelial defect, a cornea defect refractory to conventional treatments that can lead to blindness Therapeutic Mechanism KPI-012 is intended to restore normal corneal wound healing, providing a curative solution to […]

Reduced intensity conditioning with JSP191 prior to TCRαβ+ T-cell/CD19+ B-cell depleted hematopoietic stem cell transplant for Fanconi Anemia patients

Therapeutic Candidate or Device αβdepleted-HSC grafts and a reduced-intensity preparative conditioning regimen containing JSP191 Indication Treatment of patients with all genetic forms of Fanconi Anemia (FA) with evidence of cytopenias Therapeutic Mechanism Fanconi anemia (FA) patients undergoing HCT have heightened sensitivity to current conditioning regimens and short and long-term sensitivity to graft versus host disease […]